Health Economic Analysis of Islet Cell Transplantation for the Stabilization of the Severe Forms of Type 1 Diabetes

Type 1 Diabetes Clinical Trial

— STABILOT  

Official title:

Health Economic Analysis of Islet Cell Transplantation for Patients With Severe Forms of Brittle Type 1 Diabetes

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02854696
• Other study ID #: 38RC14.453
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: July 7, 2016
• Est. completion date: December 2023

Study information

• Verified date: June 2022
• Source: University Hospital, Grenoble
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The main goal is to perform a cost-utility analysis to compare islet cell transplantation versus best medical treatment (defined as Sensor augmented pump therapy) for patients with brittle type1 diabetes.

Description:

The main goal is to perform a cost-utility analysis to compare islet cell transplantation versus best medical treatment (defined as Sensor augmented insulin pump therapy) for patients with brittle type1 diabetes.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 42
• Est. completion date: December 2023
• Est. primary completion date: May 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

Inclusion Criteria :

• Suffering from diabetes since more than 5 years

• Patient with brittle type 1 diabetes despite an optimized insulin treatment and educational training will be included. A patient will be considered as experiencing a brittle type 1 diabetes if at least two criteria are present among: persistence of severe hypoglycemia, occurrence of ketoacidosis events without obvious etiology, diagnosis of unaware hypoglycemic episodes < 3 mmol/l based on CGM or self-monitoring blood glucose data, a mean blood glucose standard deviation>50%, MAGE index (Mean amplitude of glucose excursions)>60 mg/dl, LBGI index (low blood glucose index)>5, Clarke score=4 or HYPOSCORE>800.

• Insulin needs < 0,85 U/kg/day

• HbA1c < 12% ;

• No residual insulin secretion (plasmatic basal and stimulated C-peptide < 0.3 ng/ml)

• Social Security membership or benefit from Social Security

• Patients who signed the consent form

Exclusion Criteria :

Exclusion criteria related to islet infusion:

• Hemostatic disorders, pre-existing liver disease (PAL, Gamma-GT, ASAT-ALAT >2N) or vesicular lithiasis.

Exclusion criteria related to diabetic complications:

• Evolutive proliferative retinopathy, evolutive nephropathy (Glomerular filtration rate <30 ml/min/1.73m2 and/or proteinuria >0.5g/day), evolutive cardiopathy or obliterative arteriopathy with trophic cutaneous lesions.

Exclusion criteria related to immunosuppressant use:

• Hemoglobin < 110mg/dL in women and < 120 mg/dL in men, leuconeutropenia, thrombopenia, systemic infection including chronic hepatitis B, C and VIH, neoplasia disease and hypertension>160/100 mmHg.

• Corticoid treatment (except for patient that benefited from a kidney graft with maintenance steroid therapy)

• Presence of anti-HLA antibody directed against the donor

• Positive B or T cells crossmatch

Related Conditions & MeSH terms

• Diabetes Mellitus
• Diabetes Mellitus, Type 1
• Type 1 Diabetes

Intervention

Procedure:
• Islet graft
Patients will be transplanted with pancreatic islet cells

Drug:
• best medical care
Patients will continue their insulin treatment

Locations

Country	Name	City	State
France	University Hospital of Besançon	Besancon
France	university hospital of Clermont Ferrand	Clermont Ferrand
France	Grenoble University Hospital	Grenoble
France	University hospital of Lille	Lille
France	University Hospital of Lyon	Lyon
France	University Hospital of Montpellier	Montpellier
France	University hospital of Nancy	Nancy
France	university hospital of Nantes	Nantes
France	APHP	Paris
France	University hospital of Strasbourg	Strasbourg
Switzerland	University Hospital of Geneva	Geneva

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Grenoble

Countries where clinical trial is conducted

France,  Switzerland, 

References & Publications (4)

Badet L, Benhamou PY, Wojtusciszyn A, Baertschiger R, Milliat-Guittard L, Kessler L, Penfornis A, Thivolet C, Renard E, Bosco D, Morel P, Morelon E, Bayle F, Colin C, Berney T; GRAGIL Group. Expectations and strategies regarding islet transplantation: metabolic data from the GRAGIL 2 trial. Transplantation. 2007 Jul 15;84(1):89-96. — View Citation

Barton FB, Rickels MR, Alejandro R, Hering BJ, Wease S, Naziruddin B, Oberholzer J, Odorico JS, Garfinkel MR, Levy M, Pattou F, Berney T, Secchi A, Messinger S, Senior PA, Maffi P, Posselt A, Stock PG, Kaufman DB, Luo X, Kandeel F, Cagliero E, Turgeon NA, Witkowski P, Naji A, O'Connell PJ, Greenbaum C, Kudva YC, Brayman KL, Aull MJ, Larsen C, Kay TW, Fernandez LA, Vantyghem MC, Bellin M, Shapiro AM. Improvement in outcomes of clinical islet transplantation: 1999-2010. Diabetes Care. 2012 Jul;35(7):1436-45. doi: 10.2337/dc12-0063. — View Citation

Beckwith J, Nyman JA, Flanagan B, Schrover R, Schuurman HJ. A health economic analysis of clinical islet transplantation. Clin Transplant. 2012 Jan-Feb;26(1):23-33. doi: 10.1111/j.1399-0012.2011.01411.x. Epub 2011 Feb 16. — View Citation

Bellin MD, Kandaswamy R, Parkey J, Zhang HJ, Liu B, Ihm SH, Ansite JD, Witson J, Bansal-Pakala P, Balamurugan AN, Papas KK, Sutherland DE, Moran A, Hering BJ. Prolonged insulin independence after islet allotransplants in recipients with type 1 diabetes. Am J Transplant. 2008 Nov;8(11):2463-70. doi: 10.1111/j.1600-6143.2008.02404.x. Epub 2008 Sep 19. Erratum in: Am J Transplant. 2010 May;10(5):1337. Papas, K [corrected to Papas, K K]. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incremental cost- utility ratio at 1 year	The primary endpoint will be the incremental cost-effectiveness ratio at one year for islet transplantation versus Best Medical Treatment of brittle type 1 diabetes.The effectiveness will be expressed as quality adjusted life years (QALYs) in a cost-utility analysis. QALYs are a composite measure of outcomes where utilities for health states (on 0-1 scale, where 0 corresponds to death and 1 to full health) act as qualitative weights to combine quantity and quality of life. The number of QALYs in each group will be assessed with the EuroQol 5 Dimensions questionnaire (EQ5D). The EQ-5D measures health status in terms of mobility, self-care, usual activities, pain/discomfort and anxiety/depression.	1 year
Secondary	Cost-effectiveness ratio at 1 year	Assessment of the cost-effectiveness ratio at 1 year between islet cell transplantation versus best medical treatment (SAP therapy) for patients with brittle type 1 diabetes without impairment of vital prognosis. Two criteria of effectiveness will be used : the life years gained and the number of hypoglycemia	1 year
Secondary	Assessment of individual medical benefit of quality of life	Evaluate with DQOL questionnaire	6 months and 1year
Secondary	Assessment of individual medical benefit in terms of metabolic efficacy	measured from the following criteria: severe hypoglycemia, HbA1c, stimulated C-peptide, fasting glucose, insulin dose or oral diabetes, glycemic variability	6 months and 1 year
Secondary	Assessment and comparison of individual medical benefit in terms of complications of islet cell transplantation between the two groups	measured from in insulin independence, hospitalizations	6 months and 1 year
Secondary	Assessment and comparison of clinical benefit for patients with brittle type 1 diabetes with impairment of vital prognosis before and after islet cell transplantation	measured from DQOL, insulin independence, complications of islet cell transplantation	1 year
Secondary	Assessment and comparison of costs for patients with brittle type 1 diabetes with impairment of vital prognosis before and after islet cell transplantation	measured from hospitalizations	1 year
Secondary	Assessment of total cost of islet cell transplantation	Assessment of total cost of islet cell transplantation for patients with type 1 diabetes without impairment of vital prognosis, from pre-transplant period until 1 year after the last injection. Two perspectives will be used: French health care system and hospital.	1 year